The portfolio includes product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3, as well as X-linked retinitis pigmentosa (XLRP). The two companies have also created a research collaboration to study new targets for other inherited retinal diseases and expand development of adeno-associated virus (AAV) manufacturing technology.

Janssen will pay MeiraGTx, which is based in London and New York, $100 million up front in cash. Janssen will gain exclusive options to develop other preclinical treatments for other types of inherited vision loss. MeiraGTx is eligible for up to $340 million in various milestone payments, as well as royalties on sales of any projects. Janssen is paying for clinical development.

“Janssen is excited to expand our portfolio with the addition of innovative assets that have the potential to improve sight or prevent the progression to blindness in inherited retinal diseases which currently have no treatment options,” stated James List, Global Therapeutic Head, Cardiovascular & Metabolism, Janssen Research & Development. “We look forward to partnering with MeiraGTx to develop these assets and explore new potential targets for inherited retinal diseases.”

This is Janssen’s biggest investment in gene therapy so far. A year ago in January 2018, it entered a partnership with the University of Pennsylvania to develop Alzheimer’s drugs using gene therapy.

Today’s deal follows an October 2018 deal Janssen and MeiraGTx formed. In that agreement, MeiraGTx is using its riboswitch technology to engineer regulatable gene therapy constructs that encode proprietary gene sequences from Janssen. MeiraGTx indicates its “gene regulation platform is a potentially transformative technology that incorporates an on/off switch for gene expression into the gene therapy vector which can then be activated using a small molecule. In this way, gene therapies can be switched on and off according to the patients’ need and the dosing requirements of the therapy.”

Gene therapies are only now making it to the market. In 2017, the U.S. Food and Drug Administration (FDA) approved Spark Therapeutics’ Luxturna (voretigene neparvovec) for a rare type of inherited blindness. Novartis also has Zolgensma in the pipeline, which might be approved this year for spinal muscular atrophy (SMA). Other companies are working on gene therapies for hemophilia, beta-thalassemia, Duchenne muscular dystrophy (DMD) and others.

However, Xconomy notes that “the U.S. healthcare system is just beginning to grapple with how to cover the costs of these high-priced, one-time, long-lasting treatments.”

Spark’s Luxturna runs $425,000 per eye. Bluebird bio is expecting its LentiGlobin, a gene therapy for beta-thalassemia, to be approved this year, and has already discussed possible alternate pay schemes, including a five-year payment plan.

Of this new deal, Mathai Mammen, Global Head of Janssen Research & Development, stated, “This collaboration builds on Janssen’s long-standing heritage of addressing the unmet needs of patients around the world. Through this collaboration, we look forward to deepening our expertise in gene therapy and leveraging our breadth of research and development expertise to help bring forward new treatment options for people living with inherited retinal diseases.”